TARGETED GENE THERAPY
FOR
INHERITED DISEASE CONDITIONS.
ABSTRACT
Genes carry the information that determines our traits, which are features or characteristics
we inherit from our parents. Scientists believe that every human has about 20,000 to 25,000
genes. If even one of these genes is missing or has changed, it can cause disease.
Sometimes a mutation is inherited. Gene therapy uses genes to treat or prevent disease. It
may be by replacing a gene that isn’t working properly, adding a "good" gene into a person
who has a disease, or blocking a gene that is causing a problem. this chapter discusses
targeted gene therapy for some inherited diseases.
INTRODUCTION
Gene
An x-shaped chromosome is made up of tightly wound strands of DNA. DNA has smaller
sections, called genes, which can "code" for physical traits.
A gene is the basic physical and functional unit of heredity. Genes are made up of DNA.
Some genes act as instructions to make molecules called proteins. However, many genes do
not code for proteins. In humans, genes vary in size from a few hundred DNA bases to more
than 2 million bases.
1
, Genes are passed from parents to offspring and contain the information needed to
specify traits.
A gene is a region of DNA that encodes function.
A chromosome consists of a long strand of DNA containing many genes.
Genes are arranged, one after another, on structures called chromosomes.
Humans have approximately 20,000 genes arranged on their chromosomes.
Genetic disorders
A genetic disorder is a disease caused in whole or in part by a change in the DNA sequence
away from the normal sequence.
Genetic disorders can be caused by
A mutation in one gene (monogenic disorder),
2
FOR
INHERITED DISEASE CONDITIONS.
ABSTRACT
Genes carry the information that determines our traits, which are features or characteristics
we inherit from our parents. Scientists believe that every human has about 20,000 to 25,000
genes. If even one of these genes is missing or has changed, it can cause disease.
Sometimes a mutation is inherited. Gene therapy uses genes to treat or prevent disease. It
may be by replacing a gene that isn’t working properly, adding a "good" gene into a person
who has a disease, or blocking a gene that is causing a problem. this chapter discusses
targeted gene therapy for some inherited diseases.
INTRODUCTION
Gene
An x-shaped chromosome is made up of tightly wound strands of DNA. DNA has smaller
sections, called genes, which can "code" for physical traits.
A gene is the basic physical and functional unit of heredity. Genes are made up of DNA.
Some genes act as instructions to make molecules called proteins. However, many genes do
not code for proteins. In humans, genes vary in size from a few hundred DNA bases to more
than 2 million bases.
1
, Genes are passed from parents to offspring and contain the information needed to
specify traits.
A gene is a region of DNA that encodes function.
A chromosome consists of a long strand of DNA containing many genes.
Genes are arranged, one after another, on structures called chromosomes.
Humans have approximately 20,000 genes arranged on their chromosomes.
Genetic disorders
A genetic disorder is a disease caused in whole or in part by a change in the DNA sequence
away from the normal sequence.
Genetic disorders can be caused by
A mutation in one gene (monogenic disorder),
2
